Development and Validation of a Cystic Fibrosis Specific Questionnaire to Measure Abdominal Symptoms Following FDA Guidelines

What this Trial Is About

This research aims to develop, validate, and evaluate a new questionnaire called the CFAbd-Score to assess and measure abdominal symptoms in patients with cystic fibrosis, a genetic disease affecting multiple organs and shortening life expectancy. The study follows FDA guidelines to create patient-reported outcome measures PROMs specifically for cystic fibrosis and includes patients, their families, and healthcare professionals across different countries. The goal is to better understand abdominal involvement in cystic fibrosis and improve symptom tracking. The study involves developing a multimodal questionnaire assessing abdominal symptoms, evaluating its reliability, reproducibility, and responsiveness by comparing results between cystic fibrosis patients and age-matched healthy controls. It also includes creating related tools a daily symptom diary for patients and a version for children under 12 years old. The questionnaire will be adapted and tested in multiple countries and languages, with assessments of its relationship to clinical markers and ultrasound findings. Participants will complete the CFAbd-Score questionnaire and related diaries over a period of up to 7 years. The study will collect data on abdominal symptoms, genotype, clinical history, and laboratory results. The research team will monitor symptom patterns and evaluate changes related to new therapies. Healthy volunteers matched by age will also participate to provide comparison data. This long-term observational study will help refine symptom measurement tools and improve abdominal symptom assessment in cystic fibrosis care.

Development and Validation of a Disease Specific PROM to Assess Abdominal Involvement in Patients With CF (CFAbd-Score)